On September 11, 2025 enGene Holdings Inc. (Nasdaq: ENGN, "enGene" or the "Company"), a clinical-stage, non-viral gene therapy company, reported its financial results for the third quarter ended July 31, 2025, and provided a business update (Press release, enGene, SEP 11, 2025, View Source [SID1234655938]).

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"Reaching target enrollment in LEGEND’s pivotal Cohort 1 and securing RMAT designation are important milestones that mark our continued momentum," said Ron Cooper, Chief Executive Officer of enGene. "We look forward to providing a data update from the LEGEND pivotal cohort later this year. These advances bring us closer to our planned BLA filing in 2026 with the ultimate goal of delivering detalimogene to patients as a therapy designed for efficacy, safety, and ease-of-use."

Recent Corporate Updates

LEGEND study enrollment update: The Company announced that it achieved its target enrollment milestone of 100 patients with high-risk, BCG-unresponsive NMIBC carcinoma in-situ (CIS) with or without concomitant papillary disease for the pivotal cohort of its ongoing, open-label, multi-cohort Phase 2 LEGEND trial of detalimogene. Patients in the screening process remain eligible for potential enrollment in Cohort 1.

Detalimogene granted RMAT designation: The Company announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to detalimogene voraplasmid for the treatment of high-risk (HR), Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) based on previously disclosed data from the ongoing LEGEND trial. This designation provides enGene with several regulatory advantages, including early and frequent engagement with the FDA and the potential for rolling submission and priority review. Detalimogene was also granted Fast Track designation from the FDA in November 2020.

Key board and management appointments: The Company announced the appointment of Philip Astley-Sparke, William Grossman, M.D., Ph.D., and Michael Heffernan R.Ph., to its Board of Directors, and the promotion of Matthew Boyd to Chief Regulatory Officer, Jill Buck to Chief Development Officer, and Katherine Chan, M.D., M.P.H. to Executive Director, Urology Clinical Lead. Their combined experience across gene therapy, oncology, urology, clinical development, and global product launches will support enGene’s strategic transition toward its planned commercialization of detalimogene.

Anticipated Milestones

Updated preliminary data from LEGEND trial’s pivotal cohort in 4Q 2025.
Trial in progress updates from LEGEND’s additional cohorts, including HR-NMIBC patients with CIS who are naïve to treatment with BCG (Cohort 2a); HR-NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment (Cohort 2b); and BCG-unresponsive HR-NMIBC patients with papillary-only disease (Cohort 3), in 4Q 2025.
Planned BLA filing for LEGEND’s pivotal cohort in 2H 2026.
Third Quarter 2025 Financial Results

As of July 31, 2025, cash, cash equivalents and marketable securities were $224.9 million. The Company expects that its existing cash, cash equivalents and marketable securities will fund operating expenses, debt obligations and capital expenditures into 2027.

Three Months ended July 31, 2025

Total operating expenses were $29.9 million for the three months ended July 31, 2025, compared to $16.8 million for the three months ended July 31, 2024. Research and development expenses increased by $11.0 million, mainly due to increasing manufacturing and clinical costs related to the pivotal cohort of our LEGEND study and personnel-related costs. General and administrative expenses increased by $2.2 million, primarily driven by increased personnel-related expense to support the operation of a public company and increased reliance on professional services to support the Company’s preparations for potential commercialization.

For the three months ended July 31, 2025, net loss attributable to common shareholders was approximately $28.9 million, or $0.57 per share, compared to approximately $14.1 million, or $0.32 per share, for the same period for the three months ended July 31, 2024. The increase in net loss is mainly attributed to the increase in operating expenses, partially offset by net interest income earned during the period.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Non-muscle invasive bladder cancer (NMIBC) is a disease that poses a significant burden on both patients and clinics and has a massive economic impact on our healthcare system. NMIBC occurs when cancer cells grow in the tissues that line the interior of the bladder, but the cancer has not yet penetrated the muscle of the bladder wall. NMIBC can present as papillary outgrowths from the bladder wall, which are typically resected, or as carcinoma in situ (CIS), which consists of flat, multifocal lesions that cannot be resected. The two forms can also co-occur. About 75-80% of new bladder cancer diagnoses are NMIBC. Patients suffering from high-risk NMIBC who are unresponsive to the standard of care, Bacillus Calmette-Guérin (BCG), face high rates of disease recurrence (50-70%) and are potentially subject to full removal of the bladder (cystectomy) as a curative but life-altering next step.

About Detalimogene Voraplasmid

Detalimogene is a novel, investigational, non-viral gene therapy for patients with high-risk, non-muscle invasive bladder cancer (NMIBC), including Bacillus Calmette-Guérin (BCG)-unresponsive disease. It is designed to be instilled in the bladder and elicit a powerful yet localized anti-tumor immune response.

Detalimogene was developed using the Company’s Dually Derivatized Oligochitosan (DDX) platform, a technology designed to transform how gene therapies are accessed by patients and utilized by clinicians. Medicines developed with the DDX platform can potentially overcome the limitations of viral-based gene therapies, reduce complexities related to safe handling and cold storage, and streamline both manufacturing processes and administration paradigms.

Detalimogene has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration (FDA) based on its potential to address the high unmet medical need for patients with BCG-unresponsive carcinoma in situ (CIS) NMIBC with or without resected papillary tumors who are unable to undergo cystectomy. The RMAT program is intended to expedite the development and review of regenerative medicine therapies for serious or life-threatening conditions, where preliminary clinical evidence suggests potential to address unmet medical needs. Similarly, Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

About the LEGEND Trial

Detalimogene is being evaluated in the ongoing, open-label, multi-cohort, Phase 2 LEGEND trial to establish its safety and efficacy in high-risk NMIBC. LEGEND’s pivotal cohort (Cohort 1) consists of approximately 100 patients with high-risk, BCG-unresponsive NMIBC with CIS (with or without papillary disease) and is designed to serve as the basis of the Company’s planned Biologics License Application (BLA) filing. In addition to this pivotal cohort, LEGEND includes three additional cohorts, including NMIBC patients with CIS who are naïve to treatment with BCG (Cohort 2a); NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment (Cohort 2b); and BCG-unresponsive high-risk NMIBC patients with papillary-only disease (Cohort 3). The LEGEND trial is actively enrolling patients with sites participating in the USA, Canada, Europe, and the Asia-Pacific region.

On September 11, 2025 GT Medical Technologies, a medical device company dedicated to improving the lives of patients with brain tumors, reported the publication of new clinical data in the Journal of Neuro-Oncology (Press release, GT Medical Technologies, SEP 11, 2025, View Source [SID1234655937]). The study, titled "Cesium-131 collagen tile brachytherapy for salvage and recurrent intracranial metastases," was led by investigators at MD Anderson Center and supports the use of GammaTile radiation therapy for patients with recurrent brain metastases following prior tumor removal and external beam radiation therapy (EBRT).

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GammaTile is a bioabsorbable collagen implant embedded with cesium-131 radiation seeds, designed to deliver immediate, targeted radiation at the time of tumor removal surgery.1 By placing radiation directly at the site where recurrence is most likely, GammaTile provides continuous therapy while the patient recovers from surgery – closing the treatment gap that typically exists between surgery and post-operative radiation.1

Patients with recurrent brain metastases face unique challenges: prior radiation limits safe re-irradiation doses and increases the risk of radiation toxicity to healthy brain tissue, making local control difficult to achieve.2 At the same time, advancements in systemic therapies are enabling patients with metastatic disease to live longer, making local control of brain tumors more important than ever.2,3

The study evaluated 31 patients (38 tumors) previously treated with stereotactic radiosurgery (SRS) who underwent salvage resection with GammaTile. The cumulative incidence of local failure at 1 year was 13% overall, and 6.4% among tumors with gross total resection (full tumor removal) prior to GammaTile implantation.3 Interpreted in terms of local control, this translates to approximately 87% and 94% of tumors, respectively, achieving local control at 1 year, alongside a notably low incidence of symptomatic radiation toxicity (8%).3

This marks the fourth peer-reviewed publication demonstrating favorable outcomes with GammaTile in this challenging clinical scenario.2-5 Historically, recurrence rates with resection alone approach 40% at 1 year, and resection with repeat stereotactic surgery (SRS) yields 71-75% local control at 1 year, often with higher toxicity.6-9 By contrast, GammaTile has demonstrated 80-100% local control at 1 year with low levels of toxicity.2-5

This clinical evidence from MD Anderson Center builds on the growing body of data supporting GammaTile, closely following a recent study from researchers at the Miami Cancer Institute.9 That analysis demonstrated that GammaTile delivers significantly higher therapeutic dose to the surgical cavity while reducing radiation exposure to healthy brain tissue compared to stereotactic radiation therapy approaches. Together, these findings highlight both the clinical efficacy and unique dosimetric advantages of GammaTile, underscoring its potential to set a new benchmark for the management of recurrent brain metastases after prior treatment.

"Patients with recurrent brain metastases often face limited options, since prior radiation restricts what we can safely deliver and local recurrence rates remain high," said Michael Garcia, MD, MS, Chief Medical Officer, GT Medical Technologies. "These results show that GammaTile can provide durable local control with low toxicity, even in a population that has already received radiation. As systemic therapies extend survival for patients with metastatic disease, achieving reliable local brain tumor control has never been more important."

On September 11, 2025 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases, reported that it will host a virtual Investor Day on Thursday, October 2, 2025, from 10:00 a.m. and 12:00 p.m. Eastern Time (Press release, Quince Therapeutics, SEP 11, 2025, View Source [SID1234655936]). Quince’s virtual Investor Day will feature presentations from the company’s leadership team who will share the latest clinical development and corporate updates including:

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Quince Overview – Learn about the company’s pioneering Autologous Intracellular Drug Encapsulation (AIDE) technology designed to chronically deliver corticosteroids without toxicity and the potential to transform treatment paradigms across multiple rare disease indications where corticosteroids are the standard of care.
Technology Deep Dive – A look at the company’s proprietary eDSP System, which is a unique drug/device combination that uses an automated process designed to encapsulate dexamethasone sodium phosphate (eDSP) into a patient’s own red blood cells to allow for the chronic, monthly administration of corticosteroids without toxicity.
Lead Indication: Ataxia-Telangiectasia (A-T) – Insights into this devastating pediatric rare disease, encouraging prior ATTeST study results, and the company’s fully enrolled pivotal Phase 3 NEAT (Neurological Effects of eDSP on Subjects with A-T; NCT06193200/IEDAT-04-2022) clinical trial with topline results expected in the first quarter of 2026.
Mechanism of Action – Learn about multiple synergistic mechanisms of action that support eDSP’s efficacy and disease modifying activity while mitigating corticosteroid toxicity, and how transcriptomic profiling reveals novel insights and potential biomarkers in A-T.
Regulatory Overview – Discussion of the company’s Special Protocol Assessment (SPA) agreement in place with the U.S. Food and Drug Administration (FDA), orphan drug and Fast Track designations for the treatment of A-T, and the anticipated regulatory approval pathway for eDSP, assuming positive NEAT study results.
Commercial Development – Insights into the company’s attractive commercial opportunity and launch preparedness planning for eDSP, including details of its recently announced strategic relationship with Option Care Health.
Driving Value Creation – A look at the company’s strong competitive positioning, significant opportunity to quickly expand its development pipeline into additional high-value, rare disease indications like Duchenne muscular dystrophy, and its existing cash runway which provides funding through topline results and into the second quarter of 2026.
Q&A Session – Delve into the details during a Q&A session with Quince’s leadership team following the conclusion of formal presentations.
To register for this webinar, please click here. A live webcast of the presentation will be accessible on Quince’s Events page under the News & Events heading of the company’s Investor Relations website at ir.quincetx.com. An archive of the webcast will be available shortly following the end of the live event.

On September 11, 2025 Kazia Therapeutics Limited (NASDAQ: KZIA), an oncology-focused drug development company, reported new findings from a collaborative research program led by Professor Sudha Rao at QIMR Berghofer (Press release, Kazia Therapeutics, SEP 11, 2025, View Source [SID1234655934]).

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In this ex vivo study, blood samples from Stage IV HER2-positive metastatic breast cancer (mBC) patients were profiled to evaluate the effect of paxalisib, Kazia’s investigational PI3K–mTOR inhibitor, on metastatic burden. Paxalisib monotherapy demonstrated a statistically significant reduction in single circulating tumor cells and achieved a complete (100%) disruption of circulating tumor cell (CTC) clusters containing three or more cells."

Key Points
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HER2-positive breast cancer accounts for 15–20% of cases and remains a clinical challenge despite the transformative impact of HER2-targeted therapies, with many patients experiencing resistance, recurrence, or metastasis.

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Immunotherapy has demonstrated success across several solid tumors but has shown limited efficacy in HER2-positive breast cancer, underscoring the need for new therapeutic approaches.

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In this study, liquid biopsy profiling of Stage IV patients revealed that paxalisib treatment effectively disrupted CTCs and CTC clusters, which are considered biomarkers of aggressive disease and metastasis.

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Immunofluorescence analyses showed that paxalisib-treated blood samples from HER2-positive mBC patients achieved complete disruption of highly metastatic CTC clusters (≥3 cells).

"This monotherapy ex-vivo result extends our understanding of paxalisib’s potential beyond triple-negative breast cancer into HER2-positive disease," said Dr. John Friend, CEO of Kazia Therapeutics. "The ability to disrupt circulating tumor cell clusters, which are strongly associated with metastasis and poor prognosis, represents a transformative therapeutic avenue. We are particularly excited by the precision medicine aspect of this work, which leverages biomarkers to both track metastatic burden and guide therapeutic decisions. This work underscores Kazia’s commitment to expanding paxalisib’s utility across multiple subtypes of advanced breast cancer, addressing high unmet need in patients with limited options."

These findings complement Kazia’s ongoing Phase 1b trial in Stage IV triple-negative breast cancer (TNBC), where initial patient data announced in July 2025 demonstrated significant reductions in circulating tumor cells and clusters, underscoring the broader potential of paxalisib to address metastatic progression across multiple breast cancer subtypes.

Next Steps

Detailed datasets encompassing metastatic signatures and disrupted progenitor populations in Stage IV HER2-positive breast cancer have been submitted for presentation at an upcoming global oncology meeting in 2025.

On September 11, 2025 Kairos Pharma, Ltd. (NYSE American: KAPA), a clinical-stage biopharmaceutical company focused on innovative cancer therapeutics, reported that it will host a premier KOL event on Thursday, Sept. 18th at 5 p.m. ET / 2 p.m. PT to discuss diverse perspectives on the Company’s interim efficacy results from a Phase 2 trial of its lead candidate, ENV105, in treating advanced prostate cancer patients (Press release, Kairos Pharma, SEP 11, 2025, View Source [SID1234655933]). Registration is required to participate in the webcast. Interested participants can sign-up to receive the webcast link here.

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"The safety results of the trial are an important catalyst as we prepare for the announcement of our interim efficacy results in treating prostate cancer patients. This predetermined efficacy analysis occurs four months after start of combination therapy of ENV105 and apalutamide in the last safety lead-in patient. We hope to lay out the primary benefits of our compound, and to clearly demonstrate the clinical need filled by ENV105," said Dr. John Yu, CEO of Kairos Pharma. "This distinguished panel of experts will provide participants with a renewed understanding of the importance of these data and the role ENV105 can have in targeting cancer drug resistance."

Speakers in the KOL event include Dr. Neil Bhowmick, President and Chief Scientific Officer at Kairos Pharma; Dr. Umang Swami, Associate Professor in the Division of Oncology, Department of Internal Medicine at the Huntsman Cancer Institute; Dr. Richard Lee, Clinical Co-Director, The Claire and John Bertucci Center for Genitourinary Cancers at Massachusetts General Hospital, Harvard Medical School, and Dr. Edwin Posadas, Director of the Experimental Therapeutics Program and the Medical Director of the Center for Uro-Oncology Research Excellence at the Samuel Oschin Comprehensive Cancer Institute.

The interim safety analysis of the trial, announced in July of this year, demonstrated that ENV105, a first-in-class CD105 antagonist, was well tolerated when combined with standard of care hormone therapy, apalutamide, from the first 10 enrolled patients. Thus far, there have been no dose-limiting toxicities or unexpected adverse events reported to date. In addition, the treatment-related side effects were manageable with standard supportive care. Notably, no Grade 3 or 4 toxicities were observed.

With one million men in the US diagnosed with prostate cancer each year, and millions more worldwide, the development of resistance to current hormone therapies is a growing unmet need with an increasingly aging population. Castration-resistant prostate cancer refers to tumors that grow despite receiving hormone blocking agents. Treatment options remain limited after hormone therapies fail. Kairos Pharma seeks to provide a safe and effective alternative for these patients with ENV-105.